The following videos come from the annual UK MDS Forum Education Day 2016, which took place in London on 11th November 2016.

The day is targeted at all haematologists in the UK and Ireland who deal with MDS patients. Its aim is to provide information and updates on the most advanced scientific and clinical research in MDS. Please have a look at the agenda here.

We are now able to make the videos available online. Although these were initially intended for health professionals, patients and their families are also invited to watch. Our aim is to make this valuable information more widely available, not only to clinicians and nurses but also to patients, in order to educate and empower them, as well as explaining the fantastic research that goes on in MDS.

Research FOR PatientsFor an informed and empowered opinionand an improved consultationHave you made your clinical paper accessible yet?

Bone Marrow Biopsies: a less painful alternative for routine check-ups

Until now, bone marrow sampling has been the primary technique for routine follow-up checks on MDS patients after initial diagnosis. The bone marrow is the heart of the disease and reveals important clues, for example, about whether a patient is responding to therapy or whether the disease is stable or worsening (progression).

During the procedure, which can be uncomfortable, an aspiration from the patient's marrow is taken, and specific blood cells derived from the bone marrow are analysed, allowing clinicians to monitor the ongoing disease status of a patient.

More specifically, clinicians may look for the presence of particular genetic mutations within the cells, what the DNA chromosomes physically looks like (a technique broadly called cytogenetics) and the shape of certain bone marrow blood cells (morphology).

However, although necessary, bone marrow biopsies have many downsides. Most notably the stress and physical discomfort to the patient, for which some patients require sedation. It is an invasive procedure which therefore always carries a risk of infection. This risk also increases in elderly patients, or those with a low or very low neutrophil (white blood cell) count. This makes frequent sampling problematic which means patients may not be followed as closely as clinicians would like. Overall, for many patients, regular biopsies are yet another 'painful' and inconvenient aspect of living with MDS.

Peripheral Blood Sample: An alternative to biopsy

An easier alternative to a biopsy would be a peripheral blood (PB) sample (i.e. the blood already circulating in the body, which is produced in the bone marrow).

Until recently, it had not been conclusively shown in a large scale study that PB could be used to obtain similar results as a bone marrow biopsy. Also, the commonly used testing technique, called metaphase cytogenetics, does not work very well for PB samples. Therefore until now, there has been little momentum in adopting a PB sampling as standard practice.

However recent research by a group at Kings College London and the Hospital may change that (A M Mohamedali et al). Their research has demonstrated that peripheral blood samples are an equally accurate and reliable source for monitoring the genetic mutations in bone marrow derived blood cells, and hence for monitoring the disease status of a patient (please see below for full publication details).

The research group looked for the presence of various genetic abnormalities known to be frequently associated with MDS in both bone marrow samples and PB samples, and compared the results against each other.

In order to do this, they used two specific testing methods which do work for PB samples. The first is a technique called SNP – Array karyotyping (a method used to identify changes to the number of DNA strands in a cell, a feature commonly observed in MDS). The second technique used was next-generation sequencing technology (NGS) to look at over 20 genes known to harbour mutations in up to 80% of MDS patients. They found that if a gene mutation or changes to the number of DNA strands could be detected in a bone marrow biopsy sample, it could also be detected in the PB sample of the same patient. Overall, they found that the same results could be obtained for both bone marrow biopsy and PB samples using these techniques (there was a 98% concordance in results, which is extremely high).

These are very promising results which demonstrated proof of concept that PB can be used as a substitute for bone marrow biopsies. The authors of the publication recommend the use of PB for follow-up checks and believe that PB sampling has many distinct benefits over bone marrow sampling.

The most obvious being the fact that the method is less invasive and virtually pain free, with little or no risk of infection. This allows for more frequent check-ups which in turn enables closer disease monitoring for better outcomes. The procedure is also quicker and easier to perform than a biopsy, and as no sedation is required, patients are also able to leave immediately with no recovery time required.

Aside from patient benefits there are also important advantages for hospitals too. The procedure is easier and quicker to carry out than a bone marrow biopsy, therefore does not require specialist staff and cuts down on procedure time. In some cases it may even free up hospital bed time and offer cost savings.

Additionally, once the PB sample is taken, it can be analysed relatively easily using the two testing techniques described by the research group. Both the SNP – Array karyotyping and 21st century sequencing techniques were semi-automated, reliable and provided robust results, making it attractive for hospital diagnostic labs to implement.

Although a bone marrow biopsy will always be essential for initial diagnosis, finding easier, less painful, yet still accurate and reliable ways to monitor MDS patients represents a major improvement. PB sampling could spare a large population of patients the need for repeat bone marrow biopsies, making the burden of their disease a little lighter, and allowing clinicians to follow patients more closely through more regular checks.

MDS UK – Note to patients

If you are not yet offered the choice of peripheral blood (PB) sampling during routine check-ups and would like more information about its use, please contact MDS UK. This is a fairly recent technology, therefore if your haematologist has not yet started using it please hand a copy of this article to him/her. We would be happy to provide more information directly to you and/or your haematology consultant.

Clinician and Researchers Quotes

This research has provided us with the very important information that the genetic abnormalities found in the bone marrow of MDS patients are also detected in the blood. We already know that many patients acquire new genetic abnormalities during disease progression and it would therefore be possible to monitor for this on a blood sample. At present the main limiting factors for adopting this approach are the cost of these technologies as well as the complexity of analysing the data produced. The price will however fall over time and we will continue to simplify the data analysis process meaning that this has real potential for the future management of MDS patients. Unfortunately I don’t think this will replace the need for a bone marrow biopsy as this remains critical in confirming disease progression however it may allow us to detect changes early and determine when this procedure should be performed. Further research will be needed to find out if this can improve the overall management and outcome in MDS patients.

From a clinical perspective, this study is the first of its kind to demonstrate the potential use of 21st century technologies in improving the management and treatment of human diseases, especially in a disease like MDS where the majority of the patients are of old age (> 70 years). This study has clearly shown that the genetic analysis that is usually performed on bone marrow biopsy can also be reliably done on peripheral blood, thus potentially eliminating the need for repeated painful and expensive bone marrow aspirations for disease monitoring. That being said, further larger studies involving multiple centres are needed to verify these results before being introduced into routine clinical practice. Although there are challenges that need to be addressed including the cost and the data management as well as interpretation of the results, however, this technological advancement has great potential for the clinical management of MDS patients and will also help in early intervention where disease progression is suspected.

Syed Mian, PhD, Research Associate (one of the authors of this research paper) Department of Haemato-Oncology, King’s College London

Currently only a handful of specialist laboratories are equipped to perform SNP-Array karyotyping or next generation sequencing mutation analysis in MDS. The number of centres tends to be small because these types of analysis are highly specialised, require the use of expensive, dedicated equipment and require highly skilled and experienced staff. These laboratories tend to be within specialised Haematological Malignancy Diagnostic Centers such as the service in Leeds Teaching Hospitals NHS Trust and my laboratory within King’s College Hospital London. The cost of these investigations is relatively high, however the amount of genetic information obtained using these methods is much greater and results in improved certainty of diagnosis. Some of these genetic findings are also useful for informing clinicians and patients about the likely course of the disease and can also influence treatment options in a way that the conventional methods may not. Here at King’s College Hospital we have been performing this next generation sequencing mutation analysis and SNP-array karyotyping in MDS for several years. We have performed analysis on hundreds of samples and these analyses are now available as diagnostic tests. Access to these analyses make replacement of some bone marrow biopsy samples with blood a reality for our patients.

Our study was designed primarily with the patient benefit in mind. Being a tertiary referral centre for MDS, there was a clear need to improve on existing methods in aiding patient diagnosis and enable frequent follow up of patients. The data is an extension of our earlier publication in the journal Blood (2013) and confirms the very high concordance of the genetic information obtained from the bone marrow and peripheral blood. I am delighted that MDS UK has taken the initiative to disseminate this information to the community so that patients may benefit from cutting edge research tools to help and with their MDS journey

The Cancer Drug Fund (CDF)

de-listing further drugs

Many blood cancer patients are about to face more severe issues of access to treatment, following the announcement that the CDF is de-listing further drugs in order to balance its budget.

Twelve of these are blood cancer drugs.
None of the cuts affect MDS patients, but the principle is at stake – and the future funding of all cancer drugs for all cancer patients.

The Blood Cancers Alliance, an informal group of all blood cancer charities, has now written to David Cameron and Jeremy Hunt to urge them to find a suitable solution to this wholly unacceptable situation.The open letter is copied here and will be published in the Times newspaper today Wedn 4th Nov 2015.Please share it widely, including your MP.
This letter is also appearing on all websites of the Blood Cancers Alliance members.

Open letter to Rt Hon David Cameron MP and Rt Hon Jeremy Hunt MP:

Blood cancer charities urge Government for Cancer Drugs Fund solution

As an alliance of blood cancer charities, and on behalf of the 27,000 blood cancer patients and their families who have signed the petition against the delisting of life-saving drugs from the Cancer Drugs Fund (CDF), we are writing to express our concern regarding the Government’s failure on the CDF.

The Government is aware of the flawed nature of the CDF, but due to politics, has let it progress to the situation where clinically effective treatments are today being removed without a long-term solution to access.

The CDF is majorly overspent and no data has been collected on whether or not any CDF-funded treatments have actually worked. As a result of these failures 12 blood cancer drug indications, previously deemed clinically effective, are set to be removed from the Fund with no guarantee of future access.

While the CDF has improved access to cancer drugs not routinely available in NHS England, it was always intended to be a temporary solution while a long-term pricing mechanism was worked out. The proposed consultation on the CDF has taken too long to materialise, and as the new CDF system is set to be in place from April 2016, the time is rapidly decreasing for stakeholders to shape a system that is fit for purpose.

Through the revised CDF and Accelerated Access Review, there is a real opportunity to put patients at the heart of the system and ensure they are able to access the most innovative medicines. This is an opportunity that the Government can no longer afford to miss.

“On 1 April 2013, NHS England took on responsibility for the operational management of the Cancer Drugs Fund (CDF). The NHS spends approximately £1.3 billion annually on the provision of cancer drugs within routine commissioning. The CDF was established as an additional funding source to this.

The CDF has provided an additional £200m each year since then to enable patients to access drugs that would not otherwise have been routinely available from the NHS. NHS England recently pledged an additional £160m over the next two years to strengthen the fund. It was established in 2010 and will run until the end of March 2016.

There is a single, national list of drugs and indications that the CDF will routinely fund and standard operating procedures for administration of the fund.”

2015 MDS World Awareness Day: Video Stories

25/10/2015Some of our true champions for MDS awareness have shared their stories with us. Watch their video clips and share them in support of MDS World Awareness Day 2015!

Emma Paine: What happens when you need a second transplant?

Emma is an MDS patient. She had a first stem cell transplant in 2011 – and recovered well. Until now.

She is currently in hospital having chemotherapy and urgently needs a second transplant.
BUT – funding for second transplants is being revised in the UK – due to “cost-effectiveness” issues.
18 months ago, Emma would have received her potentially life-saving transplant without a problem.
Given the current NHS financial crisis, and new clinical data on the success of 2nd transplants after a relapse, the Dept of Health has tightened the rules relating to this procedure – and hospitals all over the UK need to seek special funding requests for those patients (IFR=Individual Funding Requests)

Olivia, Tilly and Ellis Hepburn make an appeal

Olivia, Tilly and Ellis, MDS super heroes and children of Lisa and Gavin Hepburn, are making an appeal for people to donate blood and stem cells to save lives of people like their Dad, who has MDS.

Lisa told us: “Today is MDS Awareness Day. Three years ago we hadn’t even heard of this disease. That’s why today is so important to make people aware. We are so grateful to the person that gave Gavin his donated bone marrow, and to the endless people that donate blood. To everyone that has helped, supported and been a shoulder to cry on these past years we are so grateful to you too…”

Their wonderful clip has already been viewed over 12,000 times on Facebook. Please keep sharing it! Thank you – on behalf of the entire MDS community.

Today is MDS Awareness Day. Three years ago we hadn’t even heard of this disease. That’s why today is so important to make people aware . We are so grateful to the person that gave Gavin his donated Bone Marrow, and to the endless people that donate blood. To everyone that has helped, supported and been a shoulder to cry on these past years we are so grateful to you too …

5 ways you can help to raise awareness about MDS

We are asking all patients, families, clinical staff, supporters to post photos or short video clips of themselves on all social media platforms, to raise awareness of this rare blood cancer MYELODYSPLASTIC SYNDROME.

All our colleagues and friends worldwide will be doing the same and will post messages online on the 25th October. See our Facebook page for more clips, and photos and the MDS Alliance website for links to all international groups marking MDS Day.

1. Record and share a video clip. You can use the message below as an example:

Hello – my name is ……. I am an MDS patient. (or I am an MDS Supporter – son/daughter/spouse/brother/sister/parent/friend/colleague/neighbour of an MDS patient). Please share my message in support of the rare blood cancer MyeloDysplastic Syndromes – and the MDS World Awareness Day on 25th October. Please support the MDS UK Patient Support Group and the international MDS Alliance. Help to register more stem cell donors and blood donors. Be a star. Thank you.

2. Share these messages of MDS patients and supporters with your family and friends

It’s #MDSWorldAwarenessDay tomorrow. Please click on my video to see how you can help! #MDSUKPatientSupportGroup

19th Congress of the European Haematology Association (EHA) has taken place this year between 12th and 15th June in Milan, Italy.

This event is set up for physicians and representatives of patient support groups to promote excellence in patient care, research and education in haematology.

As part of the Worldwide Alliance of national MDS Patient support and advocacy groups, MDS UK representative – Sophie Wintrich – attended this event to better serve the MDS patient and caregiver community.
We have provided below a brief summary of the Patient Advocacy Track and activities.
As usual, interesting MDS clinical news and updates will be provided separately and posted on our website, Facebook and our Newsletter 6.
This year Dr Wendy Ingram in Cardiff kindly agreed to write up on MDS News at EHA.

This year for the first time, the EHA congress program is complemented by a dedicated Advocacy Track. This track combines all patient and policy related sessions into one, comprehensive yet very diverse program for those interested in advocacy in haematology, patient advocacy and other related topics; and will involve:

EHA Patient Advocacy Booth (booth #206)

This is a central meeting place of all patient advocates and their peers

Patient Advocates Meeting (FRI 11:30-12:30, Room Yellow),

This is a meeting for all patient advocates in order to strengthen collaboration and links between our patient advocacy groups

This session will outline how patients can be involved as partners in clinical research and how the challenges of informed consent, trial results, and ethics in bio banking can be turned into opportunities.
EHA19 Chair Comments – Part 1 – Generics in Hamatology: The doctors’ and patients’ perspective
Jan Geissler

Old people’s diseases in young patients with chronic rare anaemias, Sexuality, fertility, family planning in haematological diseases.

ESH-EHA Joint Symposium “What do you mean, he can’t have the treatment? An interactive session for haematologists and patients” (Sat 11.45-12.45, Amber 5+6 SW2)

This renowned interactive EHA/ESH Joint Symposium will deal with how to explain to a patient that he/she can no longer receive treatment that is endorsed by national guidelines as her/his best option, or, in short: ‘what do you mean, I can’t have the treatment’?

This interactive role playing session showed the importance of doctor patient communication in consultation – as well as patient – family interaction. MDS UK gets many calls from patients who don’t feel comfortable asking too many questions, or requesting an additional opinion from an MDS expert at a Centre of Excellence. Doctor patient communication is sometimes not easy for either party, especially when it comes to difficult diagnosis – but it can be improved.

This EHA/ASH Joint Symposium will present a transnational view on the effects of the financial and economic crises, happening against a backdrop of an ageing population and an increasing prevalence of non-communicable diseases that weighs down heavily on health care budgets.
This was a really interesting session, inviting Prof JL Harousseau (ICO, France) and R Bergstroem, (EFPIA), to discuss the reasons why drug prices are so high.
Prof Harousseau is professor of haematology at the University of Nantes, France and Chairman of the French National Authority for Health (HAS). HAS can be compared to NICE, but operates more independently from Government. He was questioning why more can’t be done to lower prices by using drug volumes. He also asked why drug prices are identical, whatever the added benefit.
R Bergstroem is head of EFPIA: The European Federation of Pharmaceutical Industries and Associations represents the pharmaceutical industry operating in Europe.
He argued that Research and Development costs of past and future drugs need to be paid for, and that industry researchers are entitled to become millionaires or multi-millionaires (sic) should their drugs be effective and successfull. Marketing of those drugs also needs to be paid for.

We thought that the questions were extremely good ones, but sadly, the head of EFPIA failed to raise to the challenge to answer them in a tone and professionalism this EHA audience of doctors and patient advocates expected. Very disappointing.
The topic of drug prices is a truly difficult one – and will require a lot more flexibility from all involved to make innovative drugs available.

This is EHA’s own advocacy session highlighting EHA’s advocacy work and illustrating the importance of politics by the case of personalised medicine.

Scientific Working Group on Quality of Life (Thurs 18.30-20.00)

We welcome the addition of the Advocacy Track to the EHA congress programme and encourage our members to access the webcasts of the relevant sessions, which will be freely available to all on EHA website.

We will also post them on our website – when they become available – and open them up for comments on our on-line discussion forum.

20/06/14
We just added some photos of the EHA congress and the very successful Patient Advocacy Track, attended by many physicians and patient advocates from all over the world.

We thank the EHA Board again for their continued support, partnership and cooperation with patient advocates and look forward to EHA 2015, with some exciting new themes for the now very well established EHA Patient Advocacy Track.
We also congratulate this year’s EHA President Dr Christine Chomienne and all of the EHA teams and contributors for the most successful EHA congress to date – with an amazing 10.400 attendees.
And many thanks to patient advocate Jan Geissler from EUPATI and CML Advocates, for being such a strong and passionate spokesperson for all patient advocacy issues.
Finally thank you to all patient support group representatives and colleagues who attended EHA this year, helped man our patient advocacy booth, answered physician queries and requests and contributed to all patient advocacy sessions.
We hope to see many more of you at EHA 2015 in Vienna!